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Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
Weber L, Frati G, Felix T, Hardouin G, Casini A, Wollenschlaeger C, Meneghini V, Masson C, De Cian A, Chalumeau A, Mavilio F, Amendola M, Andre-Schmutz I, Cereseto A, El Nemer W, Concordet JP, Giovannangeli C, Cavazzana M, Miccio A. Weber L, et al. Among authors: andre schmutz i. Sci Adv. 2020 Feb 12;6(7):eaay9392. doi: 10.1126/sciadv.aay9392. Print 2020 Feb. Sci Adv. 2020. PMID: 32917636 Free PMC article.
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.
Weber L, Poletti V, Magrin E, Antoniani C, Martin S, Bayard C, Sadek H, Felix T, Meneghini V, Antoniou MN, El-Nemer W, Mavilio F, Cavazzana M, Andre-Schmutz I, Miccio A. Weber L, et al. Among authors: andre schmutz i. Mol Ther Methods Clin Dev. 2018 Aug 4;10:268-280. doi: 10.1016/j.omtm.2018.07.012. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30140714 Free PMC article.
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.
Lagresle-Peyrou C, Lefrère F, Magrin E, Ribeil JA, Romano O, Weber L, Magnani A, Sadek H, Plantier C, Gabrion A, Ternaux B, Félix T, Couzin C, Stanislas A, Tréluyer JM, Lamhaut L, Joseph L, Delville M, Miccio A, André-Schmutz I, Cavazzana M. Lagresle-Peyrou C, et al. Among authors: andre schmutz i. Haematologica. 2018 May;103(5):778-786. doi: 10.3324/haematol.2017.184788. Epub 2018 Feb 22. Haematologica. 2018. PMID: 29472357 Free PMC article. Clinical Trial.
Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination.
André-Schmutz I, Dal-Cortivo L, Six E, Kaltenbach S, Cocchiarella F, Le Chenadec J, Cagnard N, Cordier AG, Benachi A, Mandelbrot L, Azria E, Bouallag N, Luce S, Ternaux B, Reimann C, Revy P, Radford-Weiss I, Leschi C, Recchia A, Mavilio F, Cavazzana M, Blanche S. André-Schmutz I, et al. J Infect Dis. 2013 Jul 15;208(2):235-43. doi: 10.1093/infdis/jit149. Epub 2013 Apr 4. J Infect Dis. 2013. PMID: 23559464
A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells.
Delville M, Soheili T, Bellier F, Durand A, Denis A, Lagresle-Peyrou C, Cavazzana M, Andre-Schmutz I, Six E. Delville M, et al. Among authors: andre schmutz i. Mol Ther Methods Clin Dev. 2018 Aug 8;10:341-347. doi: 10.1016/j.omtm.2018.08.002. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30191160 Free PMC article.
Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice.
Soheili T, Rivière J, Ricciardelli I, Durand A, Verhoeyen E, Derrien AC, Lagresle-Peyrou C, de Saint Basile G, Cosset FL, Amrolia P, André-Schmutz I, Cavazzana M. Soheili T, et al. Blood. 2016 Dec 15;128(24):2859-2862. doi: 10.1182/blood-2016-07-729871. Epub 2016 Oct 31. Blood. 2016. PMID: 27799161 Free article. No abstract available.
63 results